If you have ALS, what is a statistically significant result?

For people with amyotrophic lateral sclerosis, which attacks the body’s motor neurons and renders a person unable to move, swallow or breathe, the search for an effective treatment has been a crushing disappointment. The only drug available for the disease, approved two decades ago, typically extends life just a few months.

Then in the fall, a small California biotech company named Genervon began extolling the benefits of GM604, its new ALS drug. In an early-stage trial with 12 patients, the results were “statistically significant,” “very robust” and “dramatic,” the company said innews releases.

According to the article, the objection to these results is that there are too few study members, but that’s the whole point of doing a statistical test – your critical value jumps due to the small sample size. So it’s not clear to me, from a philosophical point of view, that having 100 patients and doing a t-test is any different from having only 12.

If I had ALS, after looking at these results, I would be outraged if the FDA refused to provide me access to the drug.

Last year, Valor, the California ALS patient, became the only person to receive GM604 through the FDA’s “compassionate use” exemption, which allows individuals with serious illnesses to receive an experimental drug. He said his limited treatment gave him a “small but detectable” improvement in speech clarity and increased the amount of water he could hold in his mouth — a big deal, he said, because it allows him the simple pleasure of enjoying a beer at the end of the day.

How can you read that and not have tears in your eyes? ALS locks you in your body, and then slowly kills you. There is no reason why the FDA should not grant accelerated approval given these results.